Novel mRNA-Based Therapy Reduces Toxic Galactose Metabolites and Overcomes Galactose Sensitivity in a Mouse Model of Classic Galactosemia.

Balakrishnan, Bijina; An, Ding; Nguyen, Vi; DeAntonis, Christine; Martini, Paolo G V; Lai, Kent

Balakrishnan, Bijina; An, Ding; Nguyen, Vi; DeAntonis, Christine; Martini, Paolo G V; Lai, Kent.

Affiliation

Balakrishnan B; Division of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City, UT 84108, USA.
An D; Moderna, Inc., Cambridge, MA 02139, USA.
Nguyen V; Moderna, Inc., Cambridge, MA 02139, USA.
DeAntonis C; Moderna, Inc., Cambridge, MA 02139, USA.
Martini PGV; Moderna, Inc., Cambridge, MA 02139, USA. Electronic address: paolo.martini@modernatx.com.
Lai K; Division of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City, UT 84108, USA. Electronic address: kent.lai@hsc.utah.edu.

Mol Ther ; 28(1): 304-312, 2020 01 08.

Article in En | MEDLINE | ID: mdl-31604675

Subject(s)

Disease Models, Animal; Galactose/blood; Galactosemias/therapy; RNA, Messenger/administration & dosage; UTP-Hexose-1-Phosphate Uridylyltransferase/administration & dosage; Animals; Animals, Newborn; Cells, Cultured; Erythrocytes/drug effects; Erythrocytes/metabolism; Female; Fibroblasts/metabolism; Galactosemias/pathology; Galactosephosphates/metabolism; Humans; Injections, Intraperitoneal; Injections, Intravenous; Liver/drug effects; Liver/metabolism; Male; Mice; Mice, Knockout; Signal Transduction/drug effects; Transfection; Treatment Outcome; UTP-Hexose-1-Phosphate Uridylyltransferase/genetics

Key words

GALT; IEM; POI; ataxia; classic galactosemia; galactose toxicity; galactose-1 phosphate; galactose-1 phosphate uridylyltransferase; inborn errors of metabolism; mRNA therapy; primary ovarian insufficiency; speech dyspraxia

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: UTP-Hexose-1-Phosphate Uridylyltransferase / RNA, Messenger / Disease Models, Animal / Galactose / Galactosemias Type of study: Diagnostic_studies Limits: Animals / Female / Humans / Male Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2020 Type: Article Affiliation country: United States

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